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Viagra Compound May Hold Promise for Treating Fatal Genetic Disease

Researchers have identified the erectile‑dysfunction drug sildenafil as a potential therapy for Leigh syndrome, a fatal mitochondrial disorder affecting roughly one in 40,000 births. In cell models, the compound corrected mitochondrial membrane potential and normalized gene expression, while treated mice and pigs showed modest lifespan extensions. A preliminary case series of six patients reported improvements in movement and, in some cases, cognition after months to years of therapy. The team has secured orphan‑drug designation in the EU and is preparing a multi‑country trial enrolling 60‑70 participants later this year.